Rare diseases

News

Hutchmed scores with drug for rare autoimmune disease

Hutchmed is preparing to file for approval of its drug for the rare disorder wAIHA in China, after chalking up a win in a pivotal phase 2/3 trial.

News

FDA declines to approve Corcept's rare disorder drug

As the celebrations for the end of 2025 gathered pace, the FDA delivered a year-end blow to Corcept by turning down its new Cushing's drug.

News

Ultragenyx, Mereo's brittle bone drug flames out in trials

Two phase 3 trials of Ultragenyx and Mereo's sclerostin inhibitor setrusumab in osteogenesis imperfecta missed their primary efficacy objectives.

News

BioMarin ends year with $4.8bn play for Amicus

BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.

News

Kyverna rises on stiff person syndrome data

Kyverna hopes to file CAR-T therapy miv-cell for approval as the first therapy for stiff person syndrome, a rare and debilitating autoimmune disease.

News

FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

Hutchmed scores with drug for rare autoimmune disease

FDA declines to approve Corcept's rare disorder drug

Ultragenyx, Mereo's brittle bone drug flames out in trials

BioMarin ends year with $4.8bn play for Amicus

Kyverna rises on stiff person syndrome data

FDA clears first gene therapy for rare genetic disease WAS

JPM: AbbVie pays $650m upfront for RemeGen cancer drug

How to meet your physicians where they are online

Rare diseases

Editor's Picks